The document discusses value assessment frameworks for cancer therapies developed by the European Society for Medical Oncology (ESMO) and the American Society of Clinical Oncology (ASCO). It describes ESMO's Magnitude of Clinical Benefit Scale (ESMO-MCBS), which provides a relative ranking of clinical benefit for new cancer drugs. Studies applying the ESMO-MCBS found that it supported treatment decisions and aligned with reimbursement decisions. While the ESMO-MCBS provides standardized benefit assessment, it only applies to comparative studies and cannot assess single-arm trials. Overall, the ESMO-MCBS provides an unbiased and standardized approach to quantify the clinical benefit of new cancer treatments.

1. How Valuable is a Cancer
Therapy ?
Noha El Baghdady

2. European Thoughts
“With increasing budget
constraints and the need to make
trade-off s in health spending,
assessing the value of cancer
medications has taken center
stage in the discussion of drugs
and drug prices”
What about us ? 

3. ESMO and ASCO have taken steps to guide the
medical community in assessing the value of
oncology therapies by releasing their value
assessment frameworks and to facilitate the
discussion on reimbursement and payment
based on value, adapted to different countries
and regions.

4. Early 2015
The European Society for
Medical Oncology (ESMO)
The ESMO Magnitude of Clinical
Benefit Scale (ESMO-MCBS)
a relative ranking of the
magnitude of clinically
meaningful benefit

5. Shortly after ESMO-MCBS v1.0
publication
The American Society of Clinical
Oncology (ASCO)
ASCO value framework
comparisons of a new treatment regimen with
the prevailing standard of care for a specific
clinical cancer indication

6. ESMO Magnitude of Clinical
Benefit Scale (ESMO-MCBS)

7. The ESMO magnitude of clinical
benefit scale (ESMO-MCBS v1.0)
• ESMO-MCBS v1.0 has been developed only for
solid cancers.
• Given the profound differences between the
curative and palliative settings the tool is
presented in two parts.

8. ESMO Magnitude of Clinical Benefit
Scale (ESMO-MCBS)
• Based on initial recommendations from 276
members of the ESMO faculty and a team of
51 expert biostatisticians ESMO MCBS V1
developed.
• ESMO intends to apply this scale prospectively
to each new anticancer drug/intervention that
will be European Medicines Agency (EMA)
approved.

9. Drugs that obtain a high score will be
highlighted in the ESMO clinical
guidelines with the aim of prompting
rapid endorsement by health
authorities across the European Union.
ESMO Magnitude of Clinical
Benefit Scale (ESMO-MCBS)
• The ESMO tool is presented in two parts:
1.Treatments with curative intent (A,B,C)
2.Non-curative interventions (5,4,3,2,1)

10. ESMO-MCBS v1.0 Forms
(Form 1)
Evaluate
adjuvant and
other
treatments
with
“curative
intent. “
This scale is
graded A, B or
C.
For early data
demonstrating
high DFS
without
mature
survival data.

11. ESMO-MCBS v1.0 Forms
(Form 1)
• ESMO scores were obtained for the cancer
drugs from Form 1 were categorized to:
High benefit (A,B)
low benefit (C)

12. ESMO-MCBS v1.0 Forms
(Form 2)
used for studies
of new agents or
approaches in
the management
of
cancers without
curative intent.
scale is graded
5, 4, 3, 2, 1,
where grades 5
and 4
represent a
high level of
proven clinical
benefit
2a, 2b,2c
versions

13. ESMO-MCBS v1.0 Forms
(Form 2)
Forms 2a
used for
therapies
evaluated using
a primary
outcome of OS.
Forms 2b
is used for
therapies
evaluated using a
primary endpoint
of PFS or TTP.
Forms 2c
used for therapies
evaluated in non-
inferiority
(equivalence)
studies and for
studies in which
the primary
outcomes are QoL,
toxicity or response
rate (RR).

14. ESMO-MCBS v1.0 Forms
(Form 2)
• ESMO scores were obtained from form 2 for
the cancer drugs were categorized to:
High benefit (3-5)
low benefit (0-2)

15. ESMO Magnitude of Clinical Benefit
Scale (ESMO-MCBS)

16. Results of First Study Assessing
Clinical Impact of ESMO-MCBS V 1.0,
07 2016

17. The Medical University of Vienna
(MUV)
• Analysed treatments and scored trials on the
ESMO-MCBS and the resulting data was in line
with clinical experience.

18. Treatment of advanced lung
cancer at the Medical University of
Vienna
Treatment Setting MCBS
Erlotinib vs
carboplatin
(OPTIMAL, CTONG
0802)*
Zhou et al27
First-line IIIB or IV,
non-squamous,
EGFR-mutated
4
Nivolumab vs
docetaxel
(Checkmate 057)
Borghaei et al43
Second-line
squamous cell lung
cancer
5

19. Field testing of the ESMO-MCBS for the
treatment of advanced breast cancer at the
Medical University of Vienna
Treatment Setting MCBS
Trastuzumab+CT±pertuz
umab (CLEOPATRA)*
Swain et al10
Swain et al11
First-line metastatic,
HER2-positive
4
Pegylated liposomal
doxorubicin vs
conventional
doxorubicin
Brien et al22
First-line metastatic 4

20. Field-testing of the ESMO-MCBS for the
treatment of advanced colorectal cancer at
the Medical University of Vienna
Treatment Setting MCBS
FOLFIRI±cetuximab
(CRYSTAL)*
Van Cutsem et al45
First-line metastatic
stratified for KRAS
wild type
4
FOLFOX4±panitumum
ab (PRIME)*
Douillard et al46
First-line metastatic
(post hoc KRAS,
NRAS BRAF wild
type)
4

21. Field testing of the ESMO-MCBS for the
treatment of advanced gastric cancer at
the Medical University of Vienna
Treatment Setting MCBS
FOLFIRI vs ECX
Guimbaud et al64
Advanced first-line
gastric or gastro-
oesophageal
adenocarcinoma
2
Salvage
chemotherapy vs best
supportive care
Kang et al70
Second-line or third-
line gastric or gastro-
oesophageal cancer
after cisplatin/5FU
2

22. The Medical University of Vienna
Conclusions
• The MCBS is an excellent
tool for daily clinical
practice of a tertiary
referral center.
• It supports treatment
decisions based on the
clinical benefit to be
expected from a novel
approach such as
immunotherapy.

23. Results of First MENA Region practical
experience Assessing Clinical Impact
of ESMO-MCBS V 1.0, 2015

24. ISPOR 18th Annual European
Congress, Milan, Italy , 2015
• To evaluate the association
between Israel’s reimbursement
decisions ESMO-Magnitude of
Clinical Benefit Scores
• Israeli reimbursement decisions
(2013-2015) demonstrate
concordance with ESMO
magnitude of clinical benefit
scoring.

25. ISPOR 18th Annual European
Congress, Milan, Italy , 2015
• 38 cancer drugs were candidates for the 2013-
2015 National List of Health System (NLHS )
• An ESMO score was available for 19/22
approved drugs and 15/16 non-approved
drugs

28. Exceptional cases
Approved:
1.Sorafenib - advanced
thyroid carcinoma
(ESMO-2)
“unmet need” – no
alternative treatment
available.

29. Exceptional cases
Non approved:
1.Docetaxel- advanced gastric cancer (ESMO-4)
–A similar drug “paclitaxel” is already provided by
health plans, ESMO grade did not compare to
paclitaxel.

30. Limitations of the ESMO-MCBS v1.0
• The ESMO-MCBS can only be applied to
comparative research outcomes; it is
therefore not applicable when evidence of
benefit derives from single arm studies.

31. Conclusion
• ESMO MCBS is a clear and
unbiased statements
regarding the magnitude of
clinical benefit from new
therapeutic approaches.
• Also, it provides a
standardized approach for
quantifying the clinical
benefit and credible
comparison of therapeutic
effectiveness of various
cancer treatments.

32. Thank you !